E4A97 – Trial Results Summary for Patients

Title

Using High-Dose Melphalan Plus Peripheral Stem Cell Transplantation to Treat Patients with Primary Systemic Amyloidosis (A Phase II Study)

Sponsor

Eastern Cooperative Oncology Group through the NCI-sponsored Cancer Cooperative Group Program

Purpose of the Study

The purpose of this study was to evaluate the safety and effectiveness of high doses of the chemotherapy drug melphalan plus peripheral stem cell transplantation in treating patients who have primary systemic amyloidosis. Amyloidosis is a disease in which a certain type of protein is deposited in various organs, causing abnormal function. It can be caused by cancer or some other disease, or may have no known cause.

Results

The purpose of this study was to evaluate the safety and effectiveness of high doses of the chemotherapy drug melphalan plus peripheral stem cell transplantation in treating patients who have primary systemic amyloidosis. Amyloidosis is a disease in which a certain type of protein is deposited in various organs, causing abnormal function. It can be caused by cancer or some other disease, or may have no known cause.

Conclusion

A successor study (E2A01 – A Phase I Study of Amifostine Followed By High-Dose Escalation of Melphalan with Stem Cell Reconstruction for Patients with Primary Systemic Amyloidosis) is currently active and a replacement study is being planned to open early in 2007.

Start Date

July 1998

Stop Date

May 2000

Number of Participating Patients

30

Eligibility Requirements

Patients were between 18 and 70 years old, with no previous amyloidosis. They had had no more than two previous courses of chemotherapy and at least four weeks had passed since they last received treatment with melphalan.

Study Design

Patients received daily injections of a substance that encourages the body to make blood (G-CSF) starting on day one. Peripheral stem cells were collected from the patient’s bloodstream through a tube inserted in a vein starting on day five. The stem cells, collected over about 10 days, were frozen. Within 30 days after stem cell collection, patients received high dose chemotherapy using the drug melphalan, and the stem cells were returned to the bloodstream through the tube in the vein. G-CSF was given daily until white blood cell counts return to safe levels.

Side Effects

Patients experienced serious side effects, including blood abnormalities which researchers expected to see with stem cell transplantation in this group of patients. Other serious side effects included diarrhea, nausea, mouth sores, anorexia, breathing and other problems. Three patients died from treatment-related heart and lung problems and multiple organ failure. The risk of infection with stem cell transplantation is high.